Viltolarsen sodium

• CAT Number: I040118
• Molecular Formula: C244H360N113Na21O88P20
• Molecular Weight: 7386.42
• Purity: ≥95%

Viltolarsen sodium (Cat No.: I040118) is an antisense oligonucleotide therapy used to treat Duchenne muscular dystrophy (DMD), a genetic disorder caused by mutations in the dystrophin gene. Viltolarsen works by promoting exon skipping during mRNA splicing, which helps restore the production of a functional, albeit shortened, form of dystrophin in muscle cells. This helps improve muscle function and slows disease progression. Viltolarsen is specifically used in patients with DMD caused by mutations amenable to exon 53 skipping, offering a targeted therapeutic approach for this rare condition.

Reference

[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031.
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[2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449.
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